NEWS

Guidance on minimizing the impact of stability studies on gene therapy batch yield

3 July 2023
Advanced Therapy Medicinal Products
Simon Walker
Global Change Facilitator

It is recognized that gene therapy manufacturing processes result in low yields, particularly in early product development stages. Gene therapy products, however, are largely subject to many of the same regulatory requirements and expectations as other large molecule biopharmaceuticals, which are produced at much greater scales.

In terms of stability studies, this situation means that if gene therapy manufacturers adhere to common paradigms, the outcome would often be little, if any, remaining product for patients or studies to support investigational new drug applications.

This need to conserve volumes of gene therapy products is why we have published Minimizing the impact of stability testing on gene therapy batch yield.

In the paper, we outline strategies for reducing the volumes required for stability studies with the goal of conserving product for patients, while remaining compliant and delivering data on critical quality attributes across the shelf life of gene therapy products.

We also make recommendations covering both drug substance and drug product, focusing on strategies for routine stability studies. To give our work some focus, we identified and clearly defined a mock viral vector product so we could make specific recommendations for the conservation of material.

Gael Debauve, Head of Gene Therapy CMC Analytics at UCB, said, “By presenting a concrete case study and discussing the available mitigation strategies, this paper aims to provide clear guidance on how to minimize the impact of stability studies on batch yield and maximize the amount of material available for the patients.”

While its scope focuses on viral vector gene therapies, the concepts discussed may apply to other modalities within cell and gene therapies, such as ex vivo genetically modified human cells.

If you are in QC, QA, regulations, or CMC leadership, this paper will help you avoid getting lost in generic considerations that may not be relevant to your product and guide you to apply them specifically to gene therapies.

“There is a strength in having something documented to take back to your organization and validate ideas that you have had, as well as share new approaches,” said Laura Sands, Head of Regulatory Affairs at Lonza. “The paper helps the reader to think outside of the norm as there is verification that others are also doing so.”

Gene therapies do not fit the standard approaches taken for traditional biologics and there is a need to think differently. Any material we can preserve and deliver to clinical studies is always in the best interest of everyone involved, especially patients. This paper gives you the strategies to do this.

This paper is a companion to the Minimizing the impact of container closure integrity testing on gene therapy batch yield and Minimizing the impact of bioburden and sterility testing on gene therapy batch yield.

For more information, download the paper here and contact Simon Walker, Global Change Facilitator, at simon.walker@biophorum.com

Cells Under Microscope
NEWS
Control of empty, full and partially filled capsids – BioPhorum establishing an industry position on this critical quality attribute
Germs On Green Background
NEWS
Building on industry feedback to standardize plasmid release specifications
Medication
NEWS
Using a closure analysis method to reduce CGT contamination risks
Vials
NEWS
How to minimize the impact of CCIT on gene therapy batch yields
Blue & Pink Test Tubes
NEWS
A bioburden and sterility testing strategy to help you conserve product

Advanced Therapy Medicinal Products

Advanced Therapy Medicinal Products support the quest for better and faster development of cell, gene and RNA therapies​ through connecting therapy developers, contract manufacturing, and testing organizations to gain an understanding and respond to the challenges faced by the advanced therapeutics industry.

Development Group

Development Group accelerates and improves the development process of biopharmaceutical medicines for the benefit of the patient. Replacing isolation with collaboration by providing a “safe” space where subject matter experts can work on sharing solutions and best practice on emerging industry trends, implementation of new technologies and common issues whilst ensuring their intellectual property and confidentiality is protected.

Drug Substance

Drug Substance advances excellence in drug substance manufacturing and testing, acting as an industry voice to accelerate technology adoption, implement post-approval change more rapidly and increase confidence in maintaining compliance.

Fill Finish

Fill Finish accelerates development and acceptance of sustainable world-class filling and packaging operations for drug product which meets the future needs of patients. Through innovative solutions resulting from the sharing of expertise, we can overcome common performance challenges and deliver on quality and regulatory compliance.

Information Technology

Information Technology accelerates digital maturity across the ​global pharmaceutical manufacturing industry.

Quality

BioPhorum Quality provides a dedicated, safe space for quality professionals from biomanufacturers and contract organizations to coalesce and enhance effective collaboration to address shared industry challenges.

Regulatory-CMC

Regulatory CMC brings leaders together who have a common goal – to improve access to medicines through innovation in the Regulatory ecosystem. It provides a dedicated space for strategic leadership and a coordinated industry voice, to move from divergence to convergence.

Supply Chain to Patient

Supply Chain to Patient transforms the performance of global pharmaceutical clinical and commercial outbound supply chains through industry collaboration by creating transparent patient-centric, resilient and agile E2E supply chains, that consistently deliver high-quality medicines ensuring an uninterrupted supply to patients.

Supply Partner

Supply Partner is a trusted advisory group and collaboration that is the engine for driving change across the global industry for all things relevant to the inbound supply chain. Addressing the challenges of complexity, resilience, cost, sustainability, and innovation.

Sustainability

Sustainability enables the industry’s transition to a low carbon, circular future – supporting members to improve patient health while respecting the planet. We activate multi-disciplinary teams drawn from our network to deliver environmental sustainability improvements across the value chain.

Technology Strategy

Technology Strategy acts as one voice of the industry to define strategy, accelerate technology transformation and support technology translation and adoption. With a purpose of enabling the future state of flexible, resilient, sustainable end-to-end biomanufacturing.

DOWNLOAD
An industry perspective on understanding AAV capsid content variants
Needle On Yellow Background
EXTERNAL-RESOURCE
An intercompany perspective on practical experiences of predicting, optimizing and analyzing high concentration biologic therapeutic formulations
DOWNLOAD
The Drug Substance 2.0 Strategic Value Framework
Coloured dots on teal, next to vial
DOWNLOAD
BioPhorum’s holistic approach to container closure integrity
Magnifying glass over a tablet all coloured blue
DOWNLOAD
Digital Plant Maturity Model 3.0
NEWS
Inaugural BioPhorum Quality face to face – get involved
PODCAST
The next step in building regulatory as a capability to enable strategic innovation across the BioPhorum community
PODCAST
Delivering a Cell and Gene Therapy Outbound Supply Chain Pathway for the Industry
DNA and Earth on blue background
DOWNLOAD
A vision for the biopharmaceutical industry’s inbound supply chain
NEWS
A major milestone – a harmonized approach to product carbon footprint data
Technology Roadmap V 2.0
DOWNLOAD
BioPhorum Technology Roadmapping roadmap vision 2.0

Publications

Documents including best practices, vision statements and peer reviewed papers to help you take value back to your business.

Webinars

Discussing all aspects of the biopharmaceutical industry, from digitization and knowledge management to raw materials, supply chain, sustainability and more. Learn from the perspectives of those at the forefront of the industry.

Podcasts

BioPhorum Connect is our podcast series that keeps you up to date with the latest news and trends in the biopharmaceutical industry. From experienced professionals to thought leaders, these podcasts bring you the insights and perspectives of experts from around the world.

Tools

A variety of resources including design tools and user requirement specifications to help you improve and streamline your business processes.

Benchmarks and Surveys

Research conducted among the BioPhorum membership providing evidence to support change and identify best practice.

Browse all

Access a variety of free tools and papers and other resources designed to provide you with up-to-date information and insights to help you make informed decisions and maximize your success. Access our resources today and start making improving and streamlining your business processes.

WEBINAR
Bioreactivity testing in single-use system biomanufacturing
WEBINAR
Bioreactivity testing in single-use system biomanufacturing
WEBINAR
Bioreactivity testing in single-use system biomanufacturing
Magnifying glass over a tablet all coloured blue
DOWNLOAD
Digital Plant Maturity Model 3.0
WEBINAR
Bioreactivity testing in single-use system biomanufacturing